Promising Drug A 'New Paradigm' For Treating Leukemia

﻿Researchers have developed a compound that delays leukemia in mice and effectively kills leukemia cells in human tissue samples, raising hopes that the drug could lead to improved treatments in people. The researchers call it an exciting "new paradigm" for treating leukemia. The findings are published in the journal Science.

The compound works by disabling an altered cellular protein that drives one type of acute myeloid leukemia, the most common form of adult leukemia. By blocking that protein, the drug allows a cancerous cell to detect that it has problems and die, rather than continue to grow and spread. In essence, the compound blocks the cellular machinery that the cancer has highjacked.

The drug is notable because of its specificity, killing cancerous cells but not healthy cells. It hits one specific protein, and only kills the leukemia cells that have this particular altered protein in them.

In accomplishing that, the researchers have overcome a major challenge in the effort to develop a new cancer treatment. When you target a mutated protein in a cancer, you would ideally like to inhibit that mutated form of the protein but not affect the normal form of the protein that's still there. As a result, this drug does not show the toxicity and side-effects associated with the traditional chemotherapy drugs used to treat acute myeloid leukemia.

Having shown the effectiveness of the compound in mouse models and human patient samples, the researchers now want to develop it further by test it in people.